CA3117694A1 - Compounds and methods for reducing prion expression - Google Patents

Compounds and methods for reducing prion expression Download PDF

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Publication number
CA3117694A1
CA3117694A1 CA3117694A CA3117694A CA3117694A1 CA 3117694 A1 CA3117694 A1 CA 3117694A1 CA 3117694 A CA3117694 A CA 3117694A CA 3117694 A CA3117694 A CA 3117694A CA 3117694 A1 CA3117694 A1 CA 3117694A1
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Prior art keywords
nucleosides
modified
seq
sugar
linked
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Pending
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CA3117694A
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English (en)
French (fr)
Inventor
Susan M. Freier
Huynh-Hoa Bui
Hien ZHAO
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of CA3117694A1 publication Critical patent/CA3117694A1/en
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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CA3117694A 2018-11-21 2019-11-21 Compounds and methods for reducing prion expression Pending CA3117694A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862770386P 2018-11-21 2018-11-21
US62/770,386 2018-11-21
PCT/US2019/062681 WO2020106996A1 (en) 2018-11-21 2019-11-21 Compounds and methods for reducing prion expression

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Publication Number Publication Date
CA3117694A1 true CA3117694A1 (en) 2020-05-28

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ID=70774613

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CA3117694A Pending CA3117694A1 (en) 2018-11-21 2019-11-21 Compounds and methods for reducing prion expression

Country Status (14)

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US (2) US12281305B2 (https=)
EP (1) EP3884053A4 (https=)
JP (3) JP7455831B2 (https=)
KR (2) KR102941589B1 (https=)
CN (2) CN113286886B (https=)
AU (2) AU2019384181B2 (https=)
BR (1) BR112021007476A2 (https=)
CA (1) CA3117694A1 (https=)
IL (1) IL283332A (https=)
MX (1) MX2021005886A (https=)
MY (1) MY205095A (https=)
SG (1) SG11202103794QA (https=)
TW (1) TWI841633B (https=)
WO (1) WO2020106996A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
US12281305B2 (en) 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
EP4110913A4 (en) * 2020-02-28 2024-07-24 University of Massachusetts OLIGONUCLEOTIDES FOR PRNP MODULATION
AU2022213384A1 (en) * 2021-01-29 2023-07-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating huntingtin
EP4298220A1 (en) * 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
US20250243492A1 (en) * 2021-10-04 2025-07-31 Atalanta Therapeutics, Inc. Compositions and methods for treatment of prion diseases
EP4453214A4 (en) * 2021-12-22 2026-03-04 Ionis Pharmaceuticals Inc Compounds and methods for modulating glycogen synthase 1
WO2023164696A2 (en) * 2022-02-28 2023-08-31 Ionis Pharmaceuticals, Inc. Rnai agents of prion expression
WO2025137143A2 (en) * 2023-12-21 2025-06-26 Aperture Therapeutics, Inc. Compositions for modulating cd33 expression
WO2025193786A1 (en) * 2024-03-12 2025-09-18 University Of Massachusetts Oligonucleotides for prnp modulation

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